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ClinicalTrials.gov public records Last synced Oct 21, 2026, 6:29 AM EDT

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Showing 1–10 of 10 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: UX007 Clear all
No Longer Available No phase listed Expanded access

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

NCT02968953
View directory record Official record
Conditions
Glucose Transporter 1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
3 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 8, 2019 · Synced Oct 21, 2026, 6:29 AM EDT
Completed Phase 2 Interventional Results available

Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies

NCT02214160
View directory record Official record
Conditions
Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency, Carnitine-acylcarnitine Translocase (CACT) Deficiency
Interventions
UX007
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
6 Months and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
9
States / cities
San Francisco, California • Washington D.C., District of Columbia • Tampa, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 1, 2023 · Synced Oct 21, 2026, 6:29 AM EDT
Completed Phase 2 Interventional Results available

A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

NCT01886378
View directory record Official record
Conditions
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency
Interventions
UX007
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
6 Months and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
7
States / cities
Washington D.C., District of Columbia • Tampa, Florida • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2021 · Synced Oct 21, 2026, 6:29 AM EDT
Not listed Phase 2 Interventional

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

NCT02696044
View directory record Official record
Conditions
Rett Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Center for Rare Neurological Diseases, Norcross, GA
Other
Eligibility
2 Years and older · Female only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Norcross, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 17, 2020 · Synced Oct 21, 2026, 6:29 AM EDT
Terminated Phase 3 Interventional Results available

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

NCT02960217
View directory record Official record
Conditions
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Interventions
UX007, Placebo
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
6 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
4
States / cities
Aurora, Colorado • Miami, Florida • Norcross, Georgia + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2020 · Synced Oct 21, 2026, 6:29 AM EDT
Completed Phase 2 Interventional Results available

Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

NCT01993186
View directory record Official record
Conditions
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Interventions
UX007, Placebo
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year to 100 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
8
States / cities
Aurora, Colorado • Miami, Florida • Orlando, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 19, 2020 · Synced Oct 21, 2026, 6:29 AM EDT
Completed Early Phase 1 Interventional

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

NCT03665636
View directory record Official record
Conditions
Glycogen Storage Disease Type I
Interventions
Triheptanoin
Drug
Lead sponsor
Areeg El-Gharbawy
Other
Eligibility
1 Month to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 15, 2021 · Synced Oct 21, 2026, 6:29 AM EDT
Terminated Phase 2 Interventional Results available

Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

NCT02599961
View directory record Official record
Conditions
Glucose Transporter Type 1 Deficiency Syndrome
Interventions
UX007
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
5
States / cities
Aurora, Colorado • Miami, Florida • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 11, 2020 · Synced Oct 21, 2026, 6:29 AM EDT
Active, not recruiting No phase listed Observational

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

NCT04632953
View directory record Official record
Conditions
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2035
U.S. locations
13
States / cities
Phoenix, Arizona • San Francisco, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 21, 2026, 6:29 AM EDT